A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

March 28, 2025 updated by: Editas Medicine, Inc.
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
54

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • Ottawa Hospital Research Institute
      • Toronto, Ontario, Canada, M5G 2M9
        • Princess Margaret Cancer Centre-University Health Network
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Connecticut
      • New Haven, Connecticut, United States, 06511
        • Smilow Cancer Hospital
    • Florida
      • Saint Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
    • Minnesota
      • Minneapolis, Minnesota, United States, 55410
        • University of Minnesota
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical center
      • New York, New York, United States, 10032
        • Columbia University Medical Center - Department of Pediatrics
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Atrium Health
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers
    • Texas
      • Dallas, Texas, United States, 75246
        • Texas Oncology - Baylor Charles A. Sammons Cancer Center
      • Fort Worth, Texas, United States, 76104
        • Cook Children's

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)

Description

Inclusion Criteria:

  • Participant must have received an EDIT-301 infusion as part of a clinical study.
  • Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.

Exclusion Criteria:

  • Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
All participants
All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001)
Other: Safety and efficacy assessments
Assessed throughout the duration of the study.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Adverse events of special interest (AESI)
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Adverse events of interest (AEI)
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
All-cause mortality
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Adverse events (AEs) related to EDIT-301
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Serious adverse events (SAEs)
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion independence (TI) over time
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion reduction (TR) over time
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Average HbF (pg) per RBC over time for SCD participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Complete Blood Count (CBC) red cell indices assay over time for SCD participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Average HbF (pg) per F-cell over time for SCD participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
HbF and HbS concentration (g/dL) over time for SCD participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
HbF concentration (g/dL) over time for TDT participants
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion
Iron overload of TDT participants
Time Frame: up to 15 years post EDIT-301 infusion
As measured by a) Liver iron concentration (LIC) as assessed by R2* magnetic resonance imaging (MRI) for the first 3 years in this study; b) Cardiac iron concentration (CIC) as assessed by T2* MRI for the first 3 years of this study; c) Serum ferritin levels over time.
up to 15 years post EDIT-301 infusion
Proportion of TDT participants receiving iron chelation therapy over time.
Time Frame: up to 15 years post EDIT-301 infusion
up to 15 years post EDIT-301 infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Editas Medicine, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 17, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2040

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2040

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 9, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 9, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 12, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 2, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 28, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • EDIT-301-LTFU-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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